Bone Marrow Transplant
Bone marrow transplant can cure sickle cell disease for some people.
You get donated blood-making stem cells from someone else (a donor) who does not have sickle cell disease.
This lets your body make healthy red blood cells.
This can be called:
- Bone marrow transplant
- Hematopoietic cell transplant
- Stem cell transplant
If it works:
- You may be cured.
- You will likely have less pain.
- You could stop sickle cell disease medicines (example: Hydroxyurea).
- You will probably not need more blood transfusions for sickle cell disease.
- You will likely not get new organ damage from sickle cell disease, but it will not fix any organ damage you already have developed.
- You may not need to go to the hospital as often as before.
There are risks:
- It might not work for everyone.
- The chemotherapy may cause many side effects, some of which can be severe. It also causes hair loss, which should be temporary. Chemotherapy may limit your ability to have children in the future.
- The new cells can attack your body and cause new medical problems.
- Some parts of this treatment cause pain.
- Some people feel sad and alone during treatment, because it is so intense.
- It is not common, but there is a risk of cancer or death.
Bone marrow transplant does not work for everyone.
Your doctor can talk to you about the chance of it working for you.
Human leukocyte antigen (HLA) is on the surface of cells in your body.
- All the cells in your body have the same HLA type.
- Your HLA type is different from most other people.
- Your body will attack cells from someone with a different HLA type.
- This is why doctors are looking for someone with a matching HLA type.
There are 3 types of bone marrow transplant:
Fully matched bone marrow transplant
This is the current standard treatment. It was first used to cure a girl with sickle cell disease in 1983. Full match means the donor's cells have the same HLA type as your cells.
- If your sibling is not a full match, you may find a match in the donor registry. Not everyone can find a match in the registry.
- Bone marrow transplant using an unrelated donor may not be as good as a bone marrow transplant from a sibling.
- Bone marrow transplant using an unrelated donor may not be as good as a bone marrow transplant from a sibling.
Half matched (haploidentical) bone marrow transplant
This is a newer treatment which has only been used for 15 – 20 years for people with sickle cell disease. Half matched (haploidentical) means that only half of the HLA on the donor’s cells are similar to the HLA on your cells.
- This is only used when there is no fully matched donor.
- Parents are always half a match. Siblings and cousins can be half a match too.
- Doctors are still learning about half matched bone marrow transplant and trying to make it safer by studying this treatment in clinical trials.
- You may be able to undergo a half matched (haploidentical) bone marrow transplant as part of a research study.
Umbilical cord blood transplant
- This is only used when there is no full match donor.
- There can be more complications with a cord blood transplant.
- Finding matching cord blood is difficult.
There are different types of donors:
Full match, sibling.
- Full siblings have the same biological mother and biological father.
Full match, unrelated.
- Someone who volunteered in the bone marrow registry.
- People with African ancestry have about a 20% chance of finding a full match donor in the bone marrow registry. This is because fewer people with this background are in the registry and their HLA is more diverse.
Half matched (haploidentical).
- A parent is always a half match for their biological children.
- Some of your siblings can be a half match.
- Sometimes, other family members (cousins, aunts, uncles) could be a half match, but this is not common.
Umbilical cord blood.
- Umbilical cord blood can be donated by parents when their children are born.
- Umbilical cord blood is a good source of blood-making stem cells and can be used to do a transplant.
- There are cord blood banks which store donated cord blood.
- Your doctor can check if a cord blood bank has blood-making stem cells that are a good match to you.